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HA-GMAC
- Development stage
- Preclinical
- Lead developer
- CellGenTech
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Implantation
01
Overview
HA-GMAC is an autologous *ex vivo* gene therapy being developed by CellGenTech for the treatment of hemophilia A. The therapy utilizes the proprietary GMAC (Gene Medicine with Autologous Cells) platform, which involves harvesting a small amount of adipose tissue from the patient to isolate preadipocytes. These cells are then transduced *ex vivo* with a retroviral or lentiviral vector carrying the human coagulation factor VIII (FVIII) gene. The modified cells are expanded in culture to produce billions of mature, genetically modified adipocytes, which are then implanted back into the patient. Once implanted, these adipocytes act as a continuous "bio-factory," secreting functional FVIII into the systemic circulation to prevent or reduce bleeding episodes. The program is currently in preclinical development and has received support from the Japan Agency for Medical Research and Development (AMED).
- Other names
- FVIII-GMACFactor VIII Gene-Genetically Modified Adipocytes
02
Targets
F8 (Coagulation Factor VIIIa)
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